Catalyst overview
Research summary
This catalyst tracks a Approved update for AVLAYAH™ (tividenofusp alfa) (DNL310) in Hunter syndrome MPS II. The expected timing is based on the latest available company guidance and may change.
COMPASS study. 9/3/24- Successful meeting w/ FDA. 5/6/25- Completed submission of BLA. 7/7/25- BLA accepted with priority review. PDUFA target action date of January 5, 2026. 10/23/25– FDA extends BLA review to April 5, 2026. 11/06/25:- BLA review for accelerated approval is ongoing, with productive FDA engagement and commercial launch preparations on track. 01/29/26:- Continued Ph1/2 follow-up data for DNL310 to be presented at WORLDSymposium on February 5, 2026. 02/05/26:- WORLDSymposium Ph1/2 follow-up shows sustained biomarker normalization through Week 201; PDUFA April 5, 2026 unchanged. 02/26/26:- NEJM Ph1/2 results published. COMPASS Cohort A enrollment completed Dec 2025 to support confirmatory evidence. 03/25/26:- FDA granted accelerated approval for Avlayah for neurologic Hunter syndrome; Rare Pediatric Disease voucher awarded. 05/07/26:- U.S. launch underway; first commercial patients treated; COMPASS Ph2/3 confirmatory study ongoing to support global submissions. 08/06/26:- Q2 sales were $3.6M; Q3 revenue projected $10M-$12M; COMPASS ongoing.
Verification
Confirm timing and program details against the company announcement or filing.